How early should patient voices be heard in the research process?

I’m in Madrid at the ESMO Congress, “the European ASCO.” (ESMO is the European Society for Medical Oncology; ASCO is the American Society of Clinical Oncology, and its annual conference is huge, as is ESMO’s – over 18,000 attendees.)

The first slide here (click it to enlarge) points out a disconnect: the yellow triangle shows that today, patients are mostly involved toward the end of the process – after someone has decided what should be studied, and designed a trial to do that. At bottom right, in the blue band, we see that in this model the patient’s role is “doing things right.”

But the creator of this slide is German doctor Bettina Ryll (living in Sweden), whose husband died of malignant melanoma. She deeply understands both the mechanism of science and how it falls short compared to what we could easily do differently. And at bottom left she points to “doing the right thing.” And that, she points out in the purple triangle, is affected by how the earlier stages in the process affect patients: choosing the right thing can have more impact on patients.

The slide was presented by Mrs. Anastassia Negrouk, Head of International Regulatory and Intergroup Unit at EORTC – the leading European cancer research organization.

This is emerging as one of the questions in what I’m starting to call “patient emancipation.” What if informed, activated patients played an important role in deciding what scientists study??

An edgier version of that question is: why would we not involve patients? The answer I hear all the time is “Well, patients don’t understand this stuff.” And to that I respond, to paraphrase Let Patients Help: “It’s perverse to exclude people then say they don’t understand.” Or, as I’ve seen repeatedly in patient communities: “You might be surprised how much people can learn when their lives are at stake.”

Click to enlarge. Created by Anastassia Negrouk, head of the International Regulatory and Intergroup Unit at EORTC, based on Dr. Ryll’s graphic above.

That leads to the second graphic, created by Mrs. Negrouk, which goes one step earlier in the process. The graphic above is about designing a trial for a new drug. What about the question of what drug to create? Who gets to say what unmet need should be pursued??

Talk about patient emancipation – wow. What if patients got to boss the industry around, saying “We want you to study this, not that”? (I don’t know the answer – I’m just asking.)

My own slides from this session have a similar angle, but expressed differently. They’re here on Slideshare.

More about endpoints

Definition: The National Cancer Institute has the clearest explanation I’ve found: “In clinical trials, an event or outcome that can be measured objectively to determine whether the intervention being studied is beneficial.” Wikipedia adds a nice clear example about the choice of endpoint:

For example, a clinical trial investigating the ability of a medication to prevent heart attack might use chest pain as a clinical endpoint. Any patient enrolled in the trial who develops chest pain over the course of the trial, then, would be counted as having reached that clinical endpoint.

The results would ultimately reflect the fraction of patients who reached the endpoint of having developed chest pain, compared with the overall number of people enrolled [not the number of people who had heart attacks].

In that example, chest pain is a “surrogate endpoint” – not the result you ultimately want to achieve (fewer heart attacks), but something that’s easier to get at – something “that may correlate with a real clinical endpoint but doesn’t necessarily have a guaranteed relationship.” Surrogate endpoints are almost always chosen because they’re much easier and less expensive to test.

In any case it’s important to be clear about what you’re measuring, and why … and that’s where the question arises of who gets to say what researchers should pursue.

Share this:

Like this:

Comments

So important to recognize that crucial components of the research process are deciding what to study and determining what is “fundable.”

Also important to recognize that the “docs” doing research include many different health care providers and scientists. Nurses, psychologists, nutritionists, pharmacists, physicians and bench scientists. Important to have interdisciplinary research teams that include patients. Also important to recognize that these scientists are often influenced by their own patient or caregiver experiences.

Marge invokes the elephant in the room: research funding. Since so little of science is seen as funded by the average human – although NIH funding is US tax dollars at work, along with at least part of the funding for research at public universities all over ever’where – people don’t enter into the calculations unless they’re research subjects (a/k/a “meat puppets”).

This is a major part of the reason that I’d love to see a global army of Citizen Scientists – regular folks interested in scientific exploration and study of human illness. “Partnering with the non-compliant,” if you like. But that will, of course, take funding. Which is why we are stalking grants …

For a bit more context and why I believe that it is crucial that patients need to be involved in the first stages of clinical trial design, the presentation from which the first slide was taken- http://goo.gl/RKZDmM

I am working with a terrific company that is including patients’ input into the clinical trial design – Transparency Life Sciences. They have a portal on their web site that solicits input from patients and researchers which has FDA approval. This first ever crowdsourcing for trial design concept also just won them an NIH NCATS grant.THey are also incorporating telemonitoring into their trial design to benefit the patients and help reduce their travel time. check them out: http://www.transparencyls.com

I loved the stories in The Emperor of All Maladies that told how ideas about the origin of cancer and its treatment arose from various discoveries and political/social climates. I also found the political influence over cancer research to be eye-opening. Truth in Small Doses is on my Kindle waiting to be read.

As others have said, the government research funding process needs improvement. For one, the amount of government money allocated to researching a given disease seems primarily politically driven–dollars awarded are not proportional to the deaths caused by a disease, nor to the number of credible research ideas proposed for the disease. Lung cancer is the second leading cause of all deaths in the US, yet it receives far less research dollars per death than the other major cancers. Just after Congress passed the Recalcitrant Cancers Act to emphasize research on lung and pancreatic cancers, the sequestration hit; a lung cancer Specialized Program of Research Excellence (SPORE) was one of the first to have its funding cut.

For lung cancer (and possibly other diseases), the DoD recruits patients to help evaluate research proposals. But the NIH funding process for cancer research is still pretty opaque (at least to this epatient).

Some lung cancer advocacy groups raise money for research, and have medical advisers who help decide how that money will be spent. I don’t know whether patients are involved in the process, but I do know many family members of patients are involved in the organizations (there’s a shortage of healthy lung cancer epatient survivors).

Patient advocacy organizations are becoming active in clinical trial design. Several lung cancer advocacy groups were key in creating the innovative Lung-MAP Master Protocol trial for squamous cell lung cancer, which places patients in treatment arms based on tumor tissue genomic testing. The Bonnie J. Addario Lung Cancer Foundation was instrumental in creating the “Young Lungs” trial, which studies genomic causes of lung cancer in people diagnosed under age 40.

As far a patient involvement in the front end of clinical trials, there’s a great need for patient input in some aspects of clinical trial designs. In lung cancer, several trials seem to use boiler plate inclusion criteria that exclude patients who might benefit from the drug, perhaps because researchers just used “what we’ve always done.” Some cancer trials seem omit Progression Free Survival as an endpoint, when in reality living with cancer as a chronic illness is a valid near-term goal for those of us with lung cancer.

My husband has Stage 4 Renal Cell Carcinoma. At first, he was in demand for clinical trials. Then, as the disease progressed, he was cut. Seems to me they’re penalizing cancer patients for, well, having cancer.

I don’t know any specifics of your situation but I know the world of clinical trials is sadly not based on “Let’s try this on you” – the trials are designed to treat a specific kind of patient profile. If he seemed “in demand,” I’d bet my house that it was because his current status was in the sweet spot of each of those trials – something that none of us can control. A year earlier or later any one of those trials may or may not still be open.

And I’d bet my house that today his status doesn’t fit any trial that’s currently looking for patients.

My view when I was sick was that a century ago we wouldn’t even be having this conversation – for me it would have been over quickly.

It’s hard, I know. What we can strive for is to be sure we’ve tried every best option that’s available; we get no bonus points for wanting an option that’s not on the menu. (I told myself that over and over.) So the best someone can do, in my view, is get connected with a good medical center that knows all the latest, and a good patient group like SmartPatients.com.

From a clinical development research perspective it has to be as early as possible and I feel committed to include the patient’s voice in every project I am running. I am so thankful for all your engagement!